Ng Cheoklong, Peng Cheng, Wang Shangqian, Zheng Lei, Qu Le, Dong Pei, Ji Changwei, Xiao Jun, Chen Minfeng, Jia Zhankui, Zhang Tao, Hu Xiaoyi, Jing Taile, Xiong Wei, Wu Jianping, Ye Xiongjun, Li Fan, Yang Qing, Tang Qi, Zhao Juping, Wei Xuedong, Wu Jitao, Cai Wen, Zhu Guodong, Yu Zhixian, Yan Zejun, Li Chancan, Huang Zhiyang, Wang Zhenyu, Chen Yonghui, Zhang Jin, Xue Wei, Bao Yige, Qin Chao, Guo Gang, Huang Jiwei
Department of Urology, Renji Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Department of Urology, The Third Medical Centre, Chinese PLA General Hospital, Beijing, China.
Clin Genitourin Cancer. 2025 Aug;23(4):102353. doi: 10.1016/j.clgc.2025.102353. Epub 2025 Apr 16.
The effectiveness of combining immune checkpoint inhibitors (ICIs) and tyrosine kinase inhibitors (TKIs) in neoadjuvant therapy for renal cell carcinoma (RCC) remains unclear. This study aim to compare the efficacy and safety of neoadjuvant ICI plus TKI combination therapy versus TKI monotherapy in locally RCC patients.
This study included 185 cases of locally RCC disease(TanyNanyM0) receiving neoadjuvant therapy from 29 centers across China from January 2019 to Feburary 2024. Primary endpoint was the objective response rate (ORR) in all patients and patients with tumor thrombus (TT). Secondary endpoints included recurrence-free survival (RFS), overall survival (OS), surgical outcomes, and safety. Statistical analysis was performed to compare the results between 2 groups.
Combination therapy group had higher ORR compared to the TKI monotherapy group(30.9% vs. 16.3% in all patients, 34.5% vs. 15.6% in patients with TT) and pCR rate (14.8% vs. 0%). RFS rates were improved in patients with TT receiving combination therapy (P = .047). Furthermore, the combination therapy group had lower blood loss during surgery (200 mL vs. 300 mL, P = .004). The main limitation is the retrospective study design.
Neoadjuvant ICI plus TKI combination therapy showed promising efficacy and acceptable toxicity. These findings suggest that further investigation is warranted to explore the potential of this treatment approach.
免疫检查点抑制剂（ICI）与酪氨酸激酶抑制剂（TKI）联合用于肾细胞癌（RCC）新辅助治疗的有效性尚不清楚。本研究旨在比较新辅助ICI联合TKI治疗与TKI单药治疗在局部RCC患者中的疗效和安全性。
本研究纳入了2019年1月至2024年2月期间来自中国29个中心的185例接受新辅助治疗的局部RCC患者（TanyNanyM0）。主要终点是所有患者和有肿瘤血栓（TT）患者的客观缓解率（ORR）。次要终点包括无复发生存期（RFS）、总生存期（OS）、手术结果和安全性。进行统计分析以比较两组结果。
联合治疗组的ORR高于TKI单药治疗组（所有患者中分别为30.9%对16.3%，有TT的患者中分别为34.5%对15.6%），且病理完全缓解（pCR）率更高（14.8%对0%）。接受联合治疗的有TT患者的RFS率有所提高（P = 0.047）。此外，联合治疗组手术期间的失血量更低（200 mL对300 mL，P = 0.004）。主要局限性是回顾性研究设计。
新辅助ICI联合TKI治疗显示出有前景的疗效和可接受的毒性。这些发现表明有必要进一步研究以探索这种治疗方法的潜力。
